Data, Modeling,and Coordination Center for PrecISE Network

摘要 此U24应用程序的目标是建立数据、建模和协调中心（DMCC）， NHLBI的精确干预严重和/或恶化倾向哮喘（Precise）网络。的 Precise的目标是进行连续的、自适应的、II期/概念验证试验， 严重哮喘人群分层患者的干预。严重哮喘患者 异质性，因此是难以治疗的人群。精准医疗干预措施，重点是 为个别患者确定最佳治疗方法，而不是为大多数患者确定最佳治疗方法， 改善这一复杂患者群体的总体结局。Precise网络准备解决 这些问题其成功的核心将是一个创新的，前瞻性的和富有成效的协调中心 通过分享他们的愿景并提供 为Precise试验提供科学领导、组织支持和分析结果。关键要素将 加强合作和研究进展，包括经验丰富的领导，创新的设计和分析 方法（例如，序贯自适应设计、机器学习、神经网络、Q学习）， 全面的研究业务支持，最先进的数据管理系统，以及上级 行政支援我们的基础设施将提供公共和私人网站，其中包括Precise 面向公众、参与者和研究者的信息页面，常见问题，学习材料， 项目日历、通信中心和其他主要功能。我们将开发一个安全、用户友好的 先进的数据管理系统，包括基于网络的调查访问，数据输入，研究 管理（包括全面报告、生物样本跟踪和基于绩效的报销 工具）、与外部应用程序的集成以及质量控制。我们的研究运营和管理 团队将通过促进沟通（包括会议安排）为Precise提供坚实的基础 和会议记录，协作文档准备，跟踪法规遵从性，并支持摘要 和出版物准备在整个精密网络。这一综合结构将为 用于在Precise网络中执行高影响力、连续、自适应、第二阶段/概念验证试验， 这可能导致建立潜在的个性化治疗方法，用于严重的 和/或易于恶化的哮喘。

ABSTRACT The goal of this U24 application is to establish the Data, Modeling, and Coordination Center (DMCC) for the NHLBI's Precision Interventions for Severe and/or Exacerbation-Prone Asthma (PrecISE) Network. The objective of PrecISE is to conduct sequential, adaptive, phase II/proof of concept trials with precision interventions in stratified patient with severe asthma populations. Patients with severe asthma are heterogeneous, and thus a hard to treat population. Precision medicine interventions, which focus on identifying the best treatments for individual patients instead of the best treatments for the most patients, could improve overall outcomes among this complex patient population. The PrecISE Network is poised to address these issues. Central to its success will be an innovative, forward-looking, and productive Coordinating Center that brings cohesion to the participating Clinical Center investigators by sharing their visions and providing scientific leadership, organizational support and analytic results for PrecISE trials. Key elements that will enhance collaboration and research progress include experienced leadership, innovative design and analysis methodologies (e.g., sequential adaptive designs, machine learning, neural networks, Q-learning), comprehensive research operations support, a state-of-the-art data management system, and superior administrative support. Our infrastructure will provide both public and private websites which include PrecISE information pages for the public, participants, and investigators, frequently asked questions, study materials, project calendar, a communications hub, and other key features. We will develop a secure, user-friendly advanced data management system that includes web-based access for surveys, data entry, research management (including comprehensive reporting, biosample tracking and performance-based reimbursement tools), integration with external applications, and quality control. Our research operations and administration teams will provide a solid foundation to PrecISE by facilitating communication including meeting arrangements and minutes, collaborative document preparation, tracking of regulatory compliance, and support for abstract and publication preparation across the PrecISE Network. Together, this integrated structure will set the stage for the execution of high-impact, sequential, adaptive, phase II/proof of concept trials in the PrecISE Network, which can lead to the establishment of potential personalized treatment approaches for patients with severe and/or exacerbation-prone asthma.